Curing at a Cost: The Zolgensma Revolution

Shobhitha CV¹ and Ravinandan AP²*
¹Final Year B. Pharm, Sree Siddaganga College of Pharmacy, Tumkur, Karnataka, India
²Assistant Professor, Sree Siddaganga College of Pharmacy, Tumkur, Karnataka, India
*Corresponding Author: Ravinandan AP, Assistant Professor, Sree Siddaganga College of Pharmacy, Tumkur, Karnataka, India.

Published: October 16, 2024

DOI: 10.55162/MCMS.07.247

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Abstract  

Few discoveries and research in medical innovation have had the transformative potential of gene therapy. Amongst the most significant recent developments is Zolgensma, a pioneering spinal Muscular Atrophy (SMA) treatment. This infrequent but devastating genetic disorder disturbs the motor nerve cells in the spinal cord and is characterized by progressive muscle weakness and paralysis.

SMA has long been a source of despair for families worldwide; it claims the lives of many children before their second birthday. However, the advent of Zolgensma, approved by the FDA in 2019, represents a beacon of hope for families grappling with this disease, often seen as a death sentence for infants.

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